Immunologic investigations into transgene directed immune-mediated myositis following delandistrogene moxeparvovec gene therapy by Rachael A. Potter, Ida H. Moeller, Sohrab Khan, Hélène Haegel, Andreas Hollenstein, Guido Steiner, Christoph Wandel, Alexander P. Murphy, Damon R. Asher, Emanuel Palatinsky, Danielle A. Griffin, Stefanie Mason, Susan T. Iannaccone, Craig M. Zaidman, Louise R. Rodino-Klapac

Get full text

Analysis of human urinary extracellular vesicles reveals disordered renal metabolism in myotonic dystrophy type 1 by Preeti Kumari, Lauren M. Sullivan, Zhaozhi Li, E. Parker Conquest, Elizabeth Cornforth, Rojashree Jayakumar, Ningyan Hu, J. Alexander Sizemore, Brigham B. McKee, Robert R. Kitchen, Paloma González-Pérez, Constance Linville, Karla Castro, Hilda Gutierrez, Soleil Samaan, Elise L. Townsend, Basil T. Darras, Seward B. Rutkove, Susan T. Iannaccone, Paula R. Clemens, Araya Puwanant, Sudeshna Das, Thurman M. Wheeler

Get full text

BET1 variants establish impaired vesicular transport as a cause for muscular dystrophy with epilepsy by Sandra Donkervoort, Niklas Krause, Mykola Dergai, Pomi Yun, Judith Koliwer, Svetlana Gorokhova, Janelle Geist Hauserman, Beryl B Cummings, Ying Hu, Rosemarie Smith, Prech Uapinyoying, Vijay S Ganesh, Partha S Ghosh, Kristin G Monaghan, Seby L Edassery, Pia E Ferle, Sarah Silverstein, Katherine R Chao, Molly Snyder, Sara Ellingwood, Diana Bharucha‐Goebel, Susan T Iannaccone, Matteo Dal Peraro, A Reghan Foley, Jeffrey N Savas, Véronique Bolduc, Dirk Fasshauer, Carsten G Bönnemann, Michael Schwake

Get full text